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Damien Marsic
University of Florida
36Publications
12H-index
421Citations
Publications 36
Newest
Published on Dec 1, 2017in Molecular Therapy8.40
Oleksandr Kondratov1
Estimated H-index: 1
(UF: University of Florida),
Damien Marsic12
Estimated H-index: 12
(UF: University of Florida)
+ 8 AuthorsMavis Agbandje-McKenna51
Estimated H-index: 51
(UF: University of Florida)
The major drawback of the Baculovirus/Sf9 system for recombinant adeno-associated viral (rAAV) manufacturing is that most of the Bac-derived rAAV vector serotypes, with few exceptions, demonstrate altered capsid compositions and lower biological potencies. Here, we describe a new insect cell-based production platform utilizing attenuated Kozak sequence and a leaky ribosome scanning to achieve a serotype-specific modulation of AAV capsid proteins stoichiometry. By way of example, rAAV5 and rAAV9 ...
Published on Dec 1, 2017in Journal of Translational Medicine4.10
David M. Markusic16
Estimated H-index: 16
(UF: University of Florida),
Timothy C. Nichols49
Estimated H-index: 49
(UNC: University of North Carolina at Chapel Hill)
+ 6 AuthorsRoland W. Herzog49
Estimated H-index: 49
(UF: University of Florida)
Background Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. However, these pivotal investigations have also identified several challenges. For example, high vector doses are often used for hepatic gene transfer, and cytotoxic T lymphocyte responses against viral capsid may occur. Therefore, achieving therapy at reduced vector doses and other strategies to reduce capsid antigen presen...
Published on Jun 23, 2017in Investigative Ophthalmology & Visual Science3.81
Sanford L. Boye35
Estimated H-index: 35
(UF: University of Florida),
Shreyasi Choudhury6
Estimated H-index: 6
(UF: University of Florida)
+ 6 AuthorsShannon E. Boye19
Estimated H-index: 19
(UF: University of Florida)
Published on Jun 23, 2017in Investigative Ophthalmology & Visual Science3.81
Shreyasi Choudhury6
Estimated H-index: 6
(UF: University of Florida),
Damien Marsic12
Estimated H-index: 12
(UF: University of Florida)
+ 7 AuthorsShannon E. Boye19
Estimated H-index: 19
(UF: University of Florida)
Brett Palaschak3
Estimated H-index: 3
(UF: University of Florida),
Damien Marsic12
Estimated H-index: 12
(UF: University of Florida)
+ 2 AuthorsDavid M. Markusic16
Estimated H-index: 16
(UF: University of Florida)
Multiple independent adeno-associated virus (AAV) gene therapy clinical trials for hemophilia B, utilizing different AAV serotypes, have reported a vector dose-dependent loss of circulating factor IX (FIX) protein associated with capsid-specific CD8 + T cell (Cap-CD8) elimination of transduced hepatocytes. Hemophilia B patients who develop transient transaminitis and loss of FIX protein may be stabilized with the immune-suppressive (IS) drug prednisolone, but do not all recover lost FIX expressi...
Published on May 1, 2016in Molecular Therapy8.40
Yuan Lu5
Estimated H-index: 5
(UF: University of Florida),
Damien Marsic12
Estimated H-index: 12
(UF: University of Florida)
+ 9 AuthorsMargaret E. White2
Estimated H-index: 2
(UF: University of Florida)
Adding tumor specific ligands to enhance vector tumor cell interaction is the conventional concept to generate tumor targeting adeno-associated viral vector (AAV). However, it remains poorly proved whether high AAV tumor cell interaction contributes to high tumor localization in vivo following systemic delivery. Here, we conducted directed evolution selections on patient derived xenograft models using a complex AAV capsid library. Uniquely, we compared the pressure for AAV tumor cell interaction...
Published on May 1, 2016in Molecular Therapy8.40
Oleksandr Kondratov1
Estimated H-index: 1
(UF: University of Florida),
Damien Marsic12
Estimated H-index: 12
(UF: University of Florida)
+ 2 AuthorsSergei Zolotukhin33
Estimated H-index: 33
(UF: University of Florida)
Recombinant Adeno-associated virus (rAAV) vectors have emerged as one of the most versatile and successful gene therapy delivery vehicles. Even though the industry is poised for the expansion into several application areas represented by orphan diseases, a simple and scalable rAAV production technology is still lacking. We have recently developed the OneBac system to allow scalable, high-titer production of the full range of rAAV serotypes by infection of stable insect Sf9 cell lines with a sing...
Published on Jan 1, 2016in Methods of Molecular Biology
Damien Marsic12
Estimated H-index: 12
(UF: University of Florida),
Sergei Zolotukhin18
Estimated H-index: 18
(UF: University of Florida)
Directed evolution represents an attractive approach to derive AAV capsid variants capable of selectively infect specific tissue or cell targets. It involves the generation of an initial library of high complexity followed by cycles of selection during which the library is progressively enriched for target-specific variants. Each selection cycle consists of the following: reconstitution of complete AAV genomes within plasmid molecules; production of virions for which each particular capsid varia...
Published on May 1, 2015in Molecular Therapy8.40
Brett Palaschak3
Estimated H-index: 3
(UF: University of Florida),
Damien Marsic12
Estimated H-index: 12
(UF: University of Florida),
David M. Markusic16
Estimated H-index: 16
(UF: University of Florida)
Predicting immune responses directed against the AAV capsid have proven to be a challenge in the translation of liver directed AAV gene therapy into the clinic. Previously we have described the first mouse model that allows us to evaluate the potential for CD8 T cell responses directed against AAV transduced hepatocytes and to define protocols and conditions to minimize this response (Martino et al. Blood 2013). Empirical data from both AAV2 and AAV8 clinical trials for hemophilia B have suggest...
Published on May 1, 2015in Molecular Therapy8.40
Damien Marsic12
Estimated H-index: 12
(UF: University of Florida),
Sergei Zolotukhin33
Estimated H-index: 33
(UF: University of Florida)
The relatively short read lengths produced by the major Next-Gen sequencing platforms (up to 300 nt for Illumina, up to 200 nt for Ion Proton) are poorly suited for analyzing large combinatorial libraries of longer nucleotide sequences, such as those involving AAV capsid genes. Despite its much lower throughput and accuracy, the PacBio single-molecule real-time technology is currently the only option for long templates, with average read lengths of 10 to 15 kb. Using the Circular Consensus Seque...
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